Children's International Polyposis study: a randomized, double-blind, placebo-controlled study of celecoxib in children with familial adenomatous polyposis

• Published in: Clinical and experimental gastroenterology Vol. 10; p. 177
• Main Authors: Burke, Carol A, Phillips, Robin, Berger, Manuela F, Li, Chunming, Essex, Margaret Noyes, Iorga, Dinu, Lynch, Patrick M
• Format: Journal Article
• Language: English
• Published: Dove Medical Press Limited 01.01.2017
• Subjects: Analysis; Care and treatment; Celecoxib; Colonic polyps; Comparative analysis; Dosage and administration; Placebos; Prevention; Safety and security measures; Treatment outcome
• ISSN: 1178-7023; 1178-7023
• DOI: 10.2147/CEG.S12184

Objective: To evaluate the efficacy and safety of celecoxib versus placebo in the prevention and treatment of colorectal polyposis in children with familial adenomatous polyposis (FAP). Methods: In this Phase III, double-blind, randomized, placebo-controlled, multicenter trial patients aged 10-17 years with FAP were randomized to celecoxib (16 mg/kg/day) or placebo for up to 5 years. Patients underwent annual assessments, including colonoscopies, to detect the time from randomization to the earliest occurrence of [greater than or equal to]20 polyps (>2 mm in size) or colorectal malignancy. The study was terminated early due to low rate of observed endpoints combined with a lower than expected enrollment rate. Descriptive results are provided. Results: Of 106 randomized patients, 55 were treated with celecoxib (mean age 12.6 years; 52.7% female) and 51 were given placebo (mean age 12.2 years; 54.9% female). Disease progression ([greater than or equal to]20 polyps, >2 mm in size) was observed in seven (12.7%) and 13 (25.5%) patients, respectively. The median time to disease progression was 2.1 years in the celecoxib group and 1.1 years for placebo. No patient developed colorectal cancer. The rate of adverse events (AEs) was similar in both groups (75.5% and 72.9%, respectively). Three patients in the celecoxib group (none in the placebo group) experienced serious AEs. Conclusion: In children with FAP, celecoxib was a well-tolerated treatment that was associated with a lower rate of colorectal polyposis and a longer time to disease progression compared with placebo. Due to the low rate of observed endpoints, the long-term impact of these results could not be ascertained. Keywords: chemoprevention, clinical trial, adenoma, colorectal